SAT0667 TREATMENT RESPONSE CRITERIA FOR ANCA-ASSOCIATED VASCULITIS: RESULTS OF A SCOPING REVIEW
Authors: Sara Monti, Kaitlin A. Quinn, Robin Christensen, Alfred Mahr, Christian Pagnoux, Carol Langford, David Jayne, Peter Merkel, Gunnar Tomasson
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background:</h3> A comprehensive assessment of outcome measures to assess response to treatment in ANCA-associated vasculitis (AAV) is necessary to implement. <h3>Objectives:</h3> To perform a review of outcome measures to assess response to treatment in AAV which will help advance methodology for clinical trials in this disease. <h3>Methods:</h3> As part of an ongoing international project to develop response criteria, we performed a scoping review to assess the tools used as outcome measures in randomized clinical trials (RCTs) of AAV. Medline, Cochrane Central, and ClinicalTrials.gov were searched from inception until November 2018 to identify RCTs enrolling patients with granulomatosis with polyangiitis and/or microscopic polyangiitis. <h3>Results:</h3> Among the 24 RCTs included in the review (Figure 1), various versions of the Birmingham Vasculitis Activity Score (BVAS) were the most widely used instruments for disease assessment. BVAS was almost always reduced to a dichotomous variable (0 or &gt; 0) providing distinction between remission and active disease. Reduction in BVAS and/or achievement of BVAS=0 represented a main study outcome (primary or secondary endpoint) in 20/24 (83%) RCTs; 6 of these trials used the BVAS/WG. Damage, mainly assessed by the Vasculitis Damage Index (VDI), was an outcome for 14/24 (58%) RCTs. Physician global assessment and patient-reported outcomes (PROs) [measures of health-related quality of life (HRQoL) and/or patient global assessment] were assessed in 7 (29%) and 14 (58%) RCTs, respectively. Assessment of renal function or activity was a major outcome or specifically included in definitions of remission/relapse in 23/24 (96%) RCTs. Timing for outcome measure assessment differed substantially, with baseline, 6 months (15/20 RCTs), and 12 months (14/20) after enrollment being the most common time points for reporting BVAS and VDI. Assessment of disease state occurred as early as 1-4 weeks after enrollment. <h3>Conclusion:</h3> Outcome measures used in RCTs of AAV include the repeated use of vasculitis specific tools to assess disease state, but with heterogeneity in the definitions for remission/relapse and timing of assessment. Intermediate states of disease activity are currently poorly defined or evaluated. Furthermore, other important outcomes in AAV, including PROs, damage measures, and global assessments are often not included as primary or confirmatory secondary outcomes in RCTs in AAV. This review highlights the need for more homogeneous outcome assessment in RCTs for AAV and the current lack of a composite measure that integrates various endpoints relevant to physicians and patients. <h3>Disclosure of Interests: </h3> Sara Monti: None declared, Kaitlin A. Quinn: None declared, Robin Christensen Grant/research support from: AbbVie Inc, and the Oak Foundation, Speakers bureau: Roche, Alfred Mahr Consultant for: Chugai Pharma France, Speakers bureau: Roche SAS Chugai Pharma France, Christian Pagnoux: None declared, Carol Langford: None declared, David Jayne Grant/research support from: David Jayne has received research grants from Chemocentryx, GSK, Roche/Genentech and Sanofi-Genzyme. He has received consultancy fees from Astra-Zeneca, Boehringer-Ingelheim, Chemocentryx, Chugai, GSK, Infla-RX, Insmed and Takeda, Peter Merkel: None declared, Gunnar Tomasson: None declared